The iCat2, GAIN (Genomic Assessment Informs Novel Therapy) Consortium Study

NCT02520713 | Active Not Recruiting

• 1 other identifier: 15-169
• Study Type: observational
• Target: 825
• Locations: 1 country
• Sites: 12

Brief Summary

This research study is evaluating the use of specialized testing of solid tumors including sequencing. The process of performing these specialized tests is called tumor profiling. The tumor profiling may result in identifying changes in genes of the tumor that indicate that a particular therapy may have activity. This is called an individualized cancer therapy (iCat) recommendation. The results of the tumor profiling and, if applicable, the iCat recommendation will be returned.

Conditions

Pediatric Solid Tumor

Keywords

Pediatric Solid Tumor

Outcome Measures

Primary Outcomes (1)

• Overall Survival

  In pediatric patients with recurrent or refractory solid tumors who undergo successful molecular profiling, a) to describe the overall survival of patients by treatment group (iCatM, iCatUM and UM); b) to identify demographic, clinical, and genomic factors associated with overall survival from the time of relapse/progression (OSr); and c)to store tumor material, derived cell lines / xenografts, and blood samples for general sample banking and potential future research.

  18 months

Secondary Outcomes (4)

• Identification of the patient, clinical, and medication access factors associated with a) having an iCat recommendation and b) with receipt of matched targeted therapy.

  2 Years

• Determination of factors associated with response and progression-free survival time by treatment group for patients with recurrent/refractory disease and measurable/evaluable disease.

  2 Years

• Description of the frequency and range of molecular alterations in pediatric solid tumors at diagnosis and at relapse including a comparison of potentially targetable variants in paired tumor samples obtained from relapse and at initial diagnosis.

  2 Years

• Determination of whether participation in a genomics study provides psychological well-being and whether that is associated with level of genomic comprehension and with receipt of an iCat recommendation.

  2 Years

Study Arms (1)

Genetic testing and GAIN report

All enrolled patients will submit specimens for sequencing and analysis.

Genetic: Genetic testing and GAIN report

Interventions

Genetic testing and GAIN report GENETIC

All patients enrolled will submit specimens for genetic analysis. If successful the report will be reviewed to look for possible recommendations and a GAIN report will be generated regardless of possible recommendations.

Genetic testing and GAIN report

Eligibility Criteria

• Age: Up to 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

High-risk, relapsed and refractory solid tumors

You may qualify if:

• Age -- Age ≤ 30 years at time of initial qualifying solid tumor diagnosis
• Diagnosis -- Histologic diagnosis of solid malignancy (excluding brain tumors and lymphoma) that meets at least one of the following criteria:
• Refractory, defined as tumor progression after initiation of standard first line therapy without having achieved a prior partial or complete remission OR Biopsy proven residual disease at the completion of planned standard initial front-line therapy.
• Recurrent, defined as tumor progression after achieving a prior partial or complete remission
• Newly diagnosed high risk disease, defined as having an expected event free survival of \< 50% at 2 years.
• Lacks definitive diagnosis or classical genomic findings after histologic review and standard molecular testing (rare tumor group).
• Examples include (eligibility not limited to these examples):
• Histology typically associated with a fusion in which fusion is not detected.
• Ewing-like sarcoma
• Undifferentiated sarcoma
• Inflammatory myofibroblastic tumor without ALK fusion
• Infantile fibrosarcoma without NTRK fusion
• Specimen Samples
• Sufficient tumor specimen available to meet the minimum requirements for profiling from diagnosis or progression / recurrence
• \--- OR

You may not qualify if:

• No Therapy Planned
• \-- Patients who have declined further anticancer therapy will be excluded.
• Performance Status
• \-- Patients with Lansky (age \< 16 years) or Karnofsky (age ≥16 years) score \< 50 will be excluded.
• Life Expectancy -- Patients with anticipated life expectancy \< 3 months will be excluded.

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead

Study Sites (12)

University of California San Francisco

San Francisco, California, 94158, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University of Chicago Comer Children's Hospital

Chicago, Illinois, 60637, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02215, United States

Location

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, 75390, United States

Location

University of Utah Childrens Medical Center

Salt Lake City, Utah, 84113, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Tumor and Germline

MeSH Terms

Interventions

Genetic Testing

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques; Genetic Techniques; Genetic Services; Health Services; Health Care Facilities Workforce and Services; Diagnostic Services; Preventive Health Services

Study Officials

• Katherine Janeway, MD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 18, 2015
• First Posted: August 13, 2015
• Study Start: October 1, 2015
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): October 1, 2026
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (12)